Could stem cells make a real difference for autism patients and their families?

CNN has a big, high-profile new articleout today on the Duke stem cell trial for autism that has been ongoing for a while and I’m wondering how good a job CNN did on this one. Too often media over-hype stem cells and this includes for autism.

I’ve covered the Duke trial before here and here, which is run by Dr. Joanne Kurtzberg, a scholar in the translational stem cell field, and Geraldine Dawson, an autism expert (they are pictured above).

The CNN article has some balance and includes cautionary notes about not jumping to conclusions so those are positive factors in conveying the appropriate message, but on the other hand the authors, Wayne Drash and Dr. Sanjay Gupta, focus so heavily on a particular patient and family portrayed as a success story that that tips the balance to a bit of a net negative I think in terms of giving readers the wrong impression. But the included cautionary notes from Arnold Kriegstein, head of the UCSF Stem Cell Program, are helpful.

There are just so many unknowns about autism and about how stem cells might help it in terms of mechanisms. The study in question is not controlled and is open-label too, factors that complicate interpretations.

Overall, it’s a close call and this article is better than most media pieces on translation of stem cells to the clinic, but I don’t think the CNN authors were quite cautious enough about stem cells for autism in this piece.

12 Comments

It’s one I’m skeptical about…here from the CNN article:
“Kurtzberg has a hypothesis about what may be happening: that certain immune cells within the cord blood are crossing the blood-brain barrier and altering brain connectivity while also suppressing inflammation, which may exist with autism.”

Injecting cord blood without knowing how they differentiate and completely ignoring where the stems cells might have landed seems to me as a placebo both for the patient AND the researcher because open-labeled. if we could not find a definite causal factors leading to the disease and looking at how DSM-5 came to associate autism with other conditions into an Autism Spectrum Disease, it’s because autism is quite a complex disease. So, injecting cord blood cells as if it was a magical cure of that works is not in the realm science but rather that of science fiction.

“…better than nothing”???. No it isn’t. Stories like this lead to false hopes and broken dreams. One can argue that touting stem cells for incurable disease is ok, since otherwise there is no hope. Bit here, there are certainly viable things that can be done rather than believing in the stem cell fairy…this is just very very sad. You are doctors. Act as such.

MOA is indeed a valid scientific requirement for the stem cell science community and until data is peer reviewed and validated any seemingly positive results will be open to legitimate questioning and doubts will remain. This is all the more important to get across effectively to the public as patient outcomes will probably fall within a wide statistical range with some not benefiting, perhaps a few even worsening or having adverse events.

Cherry picking positive outliers to tell a hopeful message serves an audience not the science.

The article to me tried to balance the optimism in the case report and deferred to the ongoing Phase II study which was clear enough ​to me and reiterated that caution from the scientific and advocacy perspective. The video was a human interest piece.

I guess the point here is how do you relay to the public case reports of hope in medical innovation with the right degree of objectivity without having to present the entire contextual story and detailed scientific data associated with other forms of publishing?

What is the scientific rationale of this? Is autism a blood defect? Because this is what umbilical cord cells are only potentially useful for. The other major use of them is their money-making potential: talking parents into paying huge sums for buying a cord blood storage service they don’t actually need, without a clinical indication, like a hereditary leukemia.
Is it another compassionate use test based on nothing else but on a evidence- and science-free “educated guess”?
Here my article on the magic of blood stem cells, Paul is also quoted there:https://forbetterscience.com/2016/04/26/the-stem-cell-faith-healers-or-magic-inside-your-bone-marrow/

Oh, for heaven’s sake! This study is no better (or worse) than what is going on in many private stem cell clinics. Just because it is going on under the auspices of the FDA and an elite research university, it is just as premature and misleading as anything Admin has elected to berate that has been reported about private stem cell clinics, including the recent reports of blindness associated with treatments for macular degeneration.

Bias, prejudice, and ignorance can really do a number on our scientific objectivity and integrity.

And we certainly shouldn’t be judging the quality of treatment studies by news reports alone. Case in point. CNN calls this a “stem cell treatment” study; but the study principal investigator states that she thinks monocytes (not stem cells) might be responsible. Objectively and accurately based on what is actually known, it is an umbilical cord blood treatment study. Who knows, if there is a treatment effect, it might be due to acellular factors in the washed cord blood!

Unfortunately, the planned next study is very unlikely to advance understanding. A cross-over study is not a true placebo trial. It can potentially address cause-effect questions when the effects are large and the metrics are objective. With the subjective evaluations use to evaluate autism, cross-over studies may appease parents of study subjects; but they pretty much insure that clarity will not be had. Look, either do what is needed or don’t bother…you may as well be doing it in a private clinic. At least there, the volume of patients evaluated might provide sufficient statistical power to ascertain confident treatment effects related to aggregate historical data for untreated patients.

Let me also add, when you are putting live (and potentially dividing) cells into a patient, who decided that 1 year is sufficient time to judge their safety? These kids need to get through puberty before we can feel safe about cancer risk. There is much clinical experience with infusing kids with umbilical cord blood after bone marrow stem cell ablation caused by high dose chemo to eradicate cancers. Some of these kids get secondary cancers after puberty. They are thought to be due to either inherent cancer predisposition or induction by their cancer eradication treatment. However, they could also be from the rescuing cord blood cells, as the risk for some secondary cancers is higher after hematopoietic stem cell transplant.

Another worrisome point that other commenters have raised earlier: What could peripheral intravenous infusion of autologous cord blood cells possible be doing to either independently or interdependently alter cognitive function within 6 to 12 months? Magic is the wrong answer.

And my final harbinger for a mushy future ahead for this work is that the authors do not mention selection bias in their article anywhere. When you are starting with families who have privately stored cord blood samples for their kids, you will have a selection bias for research subjects with greater education, financial resources, and social support that really make a difference in how well their children excel beyond the average expectations for autism spectrum disorders.

Can someone clarify something for me. I am a bit confused on how the stem cells of the child’s umbilical cord is injected? Since the ASD is never diagnosed until the child is at least one year old, where are the stem cells obtained? I didn’t think there is a bank of stem cells in a hospital somewhere.